The FDA has approved lerodalcibep (Lerochol – Lib Therapeutics), a subcutaneously injected proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). Lerodalcibep is the fourth subcutaneously injected PCSK9-directed therapy to be approved in the US; the monoclonal antibodies alirocumab (Praluent) and evolocumab (Repatha) and the small interfering RNA inclisiran (Leqvio) were approved earlier (see Table 3).1
...- Leucovorin for Cerebral Folate Deficiency
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ISSUE
The FDA has approved use of oral leucovorin calcium (folinic acid) for treatment of cerebral folate transport deficiency in patients who have a confirmed variant in the folate receptor 1 gene (FOLR1-CFTD). It is the first drug to be approved for this extremely rare disorder.
LEUCOVORIN — Leucovorin is a biologically active form of folate that can enter the CNS even when folate transport is impaired. It has been used for many years to reduce the toxicity of folate antagonists such as methotrexate and to enhance the efficacy of 5-fluorouracil in the treatment of colorectal cancer and other malignancies.
CEREBRAL FOLATE DEFICIENCY — Cerebral folate deficiency (CFD) is characterized by low levels of folate in cerebrospinal fluid and normal folate levels outside the CNS. It can be caused by folate receptor alpha autoantibodies (FRAAs) that impair transport of folate across the blood-brain …